{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,7,22]],"date-time":"2026-07-22T11:47:46Z","timestamp":1784720866099,"version":"3.55.0"},"reference-count":48,"publisher":"Impact Journals, LLC","issue":"49","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Oncotarget"],"published-print":{"date-parts":[[2017,10,17]]},"DOI":"10.18632\/oncotarget.19976","type":"journal-article","created":{"date-parts":[[2017,8,7]],"date-time":"2017-08-07T10:34:34Z","timestamp":1502102074000},"page":"85428-85441","source":"Crossref","is-referenced-by-count":22,"title":["Generation of patient specific human neural stem cells from Niemann-Pick disease type C patient-derived fibroblasts"],"prefix":"10.18632","volume":"8","author":[{"given":"Eun-Ah","family":"Sung","sequence":"first","affiliation":[{"name":"Adult Stem Cell Research Center, College of Veterinary Medicine, Seoul National University, Seoul 08826, Republic of Korea"},{"name":"Research Institute for Veterinary Science, College of Veterinary Medicine, Seoul National University, Seoul 08826, Republic of Korea"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kyung-Rok","family":"Yu","sequence":"additional","affiliation":[{"name":"Adult Stem Cell Research Center, College of Veterinary Medicine, Seoul National University, Seoul 08826, Republic of Korea"},{"name":"Research Institute for Veterinary Science, College of Veterinary Medicine, Seoul National University, Seoul 08826, Republic of Korea"},{"name":"Current\/Present address: Hematology Branch, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, MD 20892, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ji-Hee","family":"Shin","sequence":"additional","affiliation":[{"name":"Adult Stem Cell Research Center, College of Veterinary Medicine, Seoul National University, Seoul 08826, Republic of Korea"},{"name":"Research Institute for Veterinary Science, College of Veterinary Medicine, Seoul National University, Seoul 08826, Republic of Korea"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yoojin","family":"Seo","sequence":"additional","affiliation":[{"name":"Adult Stem Cell Research Center, College of Veterinary Medicine, Seoul National University, Seoul 08826, Republic of Korea"},{"name":"Current\/Present address: Biomedical Research Institute, Pusan National University Hospital, Busan 49241, Republic of Korea"},{"name":"Current\/Present address: Pusan National University School of Medicine, Busan 49241, Republic of Korea"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hyung-Sik","family":"Kim","sequence":"additional","affiliation":[{"name":"Adult Stem Cell Research Center, College of Veterinary Medicine, Seoul National University, Seoul 08826, Republic of Korea"},{"name":"Current\/Present address: Biomedical Research Institute, Pusan National University Hospital, Busan 49241, Republic of Korea"},{"name":"Current\/Present address: Pusan National University School of Medicine, Busan 49241, Republic of Korea"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Myung Guen","family":"Koog","sequence":"additional","affiliation":[{"name":"Adult Stem Cell Research Center, College of Veterinary Medicine, Seoul National University, Seoul 08826, Republic of Korea"},{"name":"Research Institute for Veterinary Science, College of Veterinary Medicine, Seoul National University, Seoul 08826, Republic of Korea"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Insung","family":"Kang","sequence":"additional","affiliation":[{"name":"Adult Stem Cell Research Center, College of Veterinary Medicine, Seoul National University, Seoul 08826, Republic of Korea"},{"name":"Research Institute for Veterinary Science, College of Veterinary Medicine, Seoul National University, Seoul 08826, Republic of Korea"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jae-Jun","family":"Kim","sequence":"additional","affiliation":[{"name":"Adult Stem Cell Research Center, College of Veterinary Medicine, Seoul National University, Seoul 08826, Republic of Korea"},{"name":"Research Institute for Veterinary Science, College of Veterinary Medicine, Seoul National University, Seoul 08826, Republic of Korea"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Byung-Chul","family":"Lee","sequence":"additional","affiliation":[{"name":"Adult Stem Cell Research Center, College of Veterinary Medicine, Seoul National University, Seoul 08826, Republic of Korea"},{"name":"Research Institute for Veterinary Science, College of Veterinary Medicine, Seoul National University, Seoul 08826, Republic of Korea"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tae-Hoon","family":"Shin","sequence":"additional","affiliation":[{"name":"Adult Stem Cell Research Center, College of Veterinary Medicine, Seoul National University, Seoul 08826, Republic of Korea"},{"name":"Research Institute for Veterinary Science, College of Veterinary Medicine, Seoul National University, Seoul 08826, Republic of Korea"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jin Young","family":"Lee","sequence":"additional","affiliation":[{"name":"Adult Stem Cell Research Center, College of Veterinary Medicine, Seoul National University, Seoul 08826, Republic of Korea"},{"name":"Research Institute for Veterinary Science, College of Veterinary Medicine, Seoul National University, Seoul 08826, Republic of Korea"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Seunghee","family":"Lee","sequence":"additional","affiliation":[{"name":"Adult Stem Cell Research Center, College of Veterinary Medicine, Seoul National University, Seoul 08826, Republic of Korea"},{"name":"Research Institute for Veterinary Science, College of Veterinary Medicine, Seoul National University, Seoul 08826, Republic of Korea"},{"name":"Institute for Stem Cell and Regenerative Medicine in Kangstem Biotech, Biomedical Science Building, Seoul National University, Seoul 08826, Republic of Korea"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tae-Wook","family":"Kang","sequence":"additional","affiliation":[{"name":"Adult Stem Cell Research Center, College of Veterinary Medicine, Seoul National University, Seoul 08826, Republic of Korea"},{"name":"Research Institute for Veterinary Science, College of Veterinary Medicine, Seoul National University, Seoul 08826, Republic of Korea"},{"name":"Institute for Stem Cell and Regenerative Medicine in Kangstem Biotech, Biomedical Science Building, Seoul National University, Seoul 08826, Republic of Korea"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Soon Won","family":"Choi","sequence":"additional","affiliation":[{"name":"Adult Stem Cell Research Center, College of Veterinary Medicine, Seoul National University, Seoul 08826, Republic of Korea"},{"name":"Research Institute for Veterinary Science, College of Veterinary Medicine, Seoul National University, Seoul 08826, Republic of Korea"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kyung-Sun","family":"Kang","sequence":"additional","affiliation":[{"name":"Adult Stem Cell Research Center, College of Veterinary Medicine, Seoul National University, Seoul 08826, Republic of Korea"},{"name":"Research Institute for Veterinary Science, College of Veterinary Medicine, Seoul National University, Seoul 08826, Republic of Korea"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"7892","published-online":{"date-parts":[[2017,8,7]]},"reference":[{"key":"1","doi-asserted-by":"crossref","first-page":"228","DOI":"10.1126\/science.277.5323.228","article-title":"Niemann-Pick C1 disease gene: homology to mediators of cholesterol homeostasis","volume":"277","author":"Sturley","year":"1997","journal-title":"Science"},{"key":"2","doi-asserted-by":"crossref","first-page":"8229","DOI":"10.1074\/jbc.M708735200","article-title":"Niemann-Pick type C1 I1061T mutant encodes a functional protein that is selected for endoplasmic reticulum-associated degradation due to protein misfolding","volume":"283","author":"Ory","year":"2008","journal-title":"J Biol Chem"},{"key":"3","doi-asserted-by":"crossref","first-page":"3411","DOI":"10.1016\/S0021-9258(18)69086-6","article-title":"Type C Niemann-Pick disease. Lysosomal accumulation and defective intracellular mobilization of low density lipoprotein cholesterol","volume":"263","author":"Pentchev","year":"1988","journal-title":"J Biol Chem"},{"key":"4","doi-asserted-by":"crossref","first-page":"16","DOI":"10.1186\/1750-1172-5-16","article-title":"Niemann-Pick disease type C.","volume":"5","author":"Vanier","year":"2010","journal-title":"Orphanet J Rare Dis"},{"key":"5","doi-asserted-by":"crossref","first-page":"152","DOI":"10.1016\/j.ymgme.2009.06.008","article-title":"Recommendations on the diagnosis and management of Niemann-Pick disease type C.","volume":"98","author":"Patterson","year":"2009","journal-title":"Mol Genet Metab"},{"key":"6","doi-asserted-by":"crossref","first-page":"2377","DOI":"10.1073\/pnas.0810895106","article-title":"Reversal of defective lysosomal transport in NPC disease ameliorates liver dysfunction and neurodegeneration in the npc1-\/- mouse","volume":"106","author":"Dietschy","year":"2009","journal-title":"Proc Natl Acad Sci U S A"},{"key":"7","doi-asserted-by":"crossref","first-page":"e6951","DOI":"10.1371\/journal.pone.0006951","article-title":"Chronic cyclodextrin treatment of murine Niemann-Pick C disease ameliorates neuronal cholesterol and glycosphingolipid storage and disease progression","volume":"4","author":"Walkley","year":"2009","journal-title":"PLoS One"},{"key":"8","doi-asserted-by":"crossref","first-page":"5620","DOI":"10.1073\/pnas.1014890108","article-title":"Histone deacetylase inhibitor treatment dramatically reduces cholesterol accumulation in Niemann-Pick type C1 mutant human fibroblasts","volume":"108","author":"Maxfield","year":"2011","journal-title":"Proc Natl Acad Sci U S A"},{"key":"9","doi-asserted-by":"crossref","first-page":"593","DOI":"10.1016\/j.bbrc.2007.06.116","article-title":"Defective cholesterol traffic and neuronal differentiation in neural stem cells of Niemann-Pick type C disease improved by valproic acid, a histone deacetylase inhibitor","volume":"360","author":"Kang","year":"2007","journal-title":"Biochem Biophys Res Commun"},{"key":"10","doi-asserted-by":"crossref","first-page":"688","DOI":"10.1007\/s13311-013-0217-2","article-title":"Treatment of Niemann--pick type C disease by histone deacetylase inhibitors","volume":"10","author":"Wiest","year":"2013","journal-title":"Neurotherapeutics"},{"key":"11","doi-asserted-by":"crossref","first-page":"292","DOI":"10.1634\/stemcells.2005-0221","article-title":"NPC1 gene deficiency leads to lack of neural stem cell self-renewal and abnormal differentiation through activation of p38 mitogen-activated protein kinase signaling","volume":"24","author":"Kang","year":"2006","journal-title":"Stem Cells"},{"key":"12","doi-asserted-by":"crossref","first-page":"686","DOI":"10.1038\/cr.2008.48","article-title":"Impaired functions of neural stem cells by abnormal nitric oxide-mediated signaling in anmodel of Niemann-Pick type C disease","volume":"18","author":"Kang","year":"2008","journal-title":"Cell Res"},{"key":"13","doi-asserted-by":"crossref","first-page":"387","DOI":"10.2217\/clp.10.19","article-title":"Niemann-Pick type C pathogenesis and treatment: from statins to sugars","volume":"5","author":"Sturley","year":"2010","journal-title":"Clin Lipidol"},{"key":"14","doi-asserted-by":"crossref","first-page":"e48561","DOI":"10.1371\/journal.pone.0048561","article-title":"Quantitative comparison of the efficacy of various compounds in lowering intracellular cholesterol levels in Niemann-Pick type C fibroblasts","volume":"7","author":"Goodson","year":"2012","journal-title":"PLoS One"},{"key":"15","doi-asserted-by":"crossref","first-page":"34","DOI":"10.1186\/1750-1172-8-34","article-title":"A human neuronal model of Niemann Pick C disease developed from stem cells isolated from patient\u2019s skin","volume":"8","author":"Beltrami","year":"2013","journal-title":"Orphanet J Rare Dis"},{"key":"16","doi-asserted-by":"crossref","first-page":"2651","DOI":"10.1093\/hmg\/dds090","article-title":"Disruption and therapeutic rescue of autophagy in a human neuronal model of Niemann Pick type C1","volume":"21","author":"Goldstein","year":"2012","journal-title":"Hum Mol Genet"},{"key":"17","doi-asserted-by":"crossref","first-page":"144","DOI":"10.1186\/1750-1172-8-144","article-title":"Niemann-Pick type C1 patient-specific induced pluripotent stem cells display disease specific hallmarks","volume":"8","author":"Frech","year":"2013","journal-title":"Orphanet J Rare Dis"},{"key":"18","doi-asserted-by":"crossref","first-page":"230","DOI":"10.5966\/sctm.2014-0127","article-title":"Rescue of anneuron phenotype identified in Niemann-Pick disease, type C1 induced pluripotent stem cell-derived neurons by modulating the WNT pathway and calcium signaling","volume":"4","author":"Malik","year":"2015","journal-title":"Stem Cells Transl Med"},{"key":"19","doi-asserted-by":"crossref","first-page":"1075","DOI":"10.1002\/stem.1917","article-title":"HPGCD outperforms HPBCD as a potential treatment for Niemann-Pick disease type C during disease modeling with iPS cells","volume":"33","author":"Era","year":"2015","journal-title":"Stem Cells"},{"key":"20","doi-asserted-by":"crossref","first-page":"866","DOI":"10.1016\/j.stemcr.2014.03.014","article-title":"Genetic and chemical correction of cholesterol accumulation and impaired autophagy in hepatic and neural cells derived from Niemann-Pick Type C patient-specific iPS cells","volume":"2","author":"Jaenisch","year":"2014","journal-title":"Stem Cell Rep"},{"key":"21","doi-asserted-by":"crossref","first-page":"5514","DOI":"10.1038\/ncomms6514","article-title":"Pathological roles of the VEGF\/SphK pathway in Niemann-Pick type C neurons","volume":"5","author":"He","year":"2014","journal-title":"Nat Commun"},{"key":"22","doi-asserted-by":"crossref","first-page":"e2147","DOI":"10.1038\/cddis.2016.16","article-title":"Necroptosis in Niemann-Pick disease, type C1: a potential therapeutic target","volume":"7","author":"Porter","year":"2016","journal-title":"Cell Death Dis"},{"key":"23","doi-asserted-by":"crossref","first-page":"1035","DOI":"10.1038\/nature08797","article-title":"Direct conversion of fibroblasts to functional neurons by defined factors","volume":"463","author":"Wernig","year":"2010","journal-title":"Nature"},{"key":"24","doi-asserted-by":"crossref","first-page":"220","DOI":"10.1038\/nature10202","article-title":"Induction of human neuronal cells by defined transcription factors","volume":"476","author":"Wernig","year":"2011","journal-title":"Nature"},{"key":"25","doi-asserted-by":"crossref","first-page":"375","DOI":"10.1016\/j.cell.2010.07.002","article-title":"Direct reprogramming of fibroblasts into functional cardiomyocytes by defined factors","volume":"142","author":"Srivastava","year":"2010","journal-title":"Cell"},{"key":"26","doi-asserted-by":"crossref","first-page":"386","DOI":"10.1038\/nature10116","article-title":"Induction of functional hepatocyte-like cells from mouse fibroblasts by defined factors","volume":"475","author":"Hui","year":"2011","journal-title":"Nature"},{"key":"27","doi-asserted-by":"crossref","first-page":"205","DOI":"10.1016\/j.stem.2013.05.024","article-title":"Induction of a hemogenic program in mouse fibroblasts","volume":"13","author":"Moore","year":"2013","journal-title":"Cell Stem Cell"},{"key":"28","doi-asserted-by":"crossref","first-page":"465","DOI":"10.1016\/j.stem.2012.02.021","article-title":"Direct reprogramming of fibroblasts into neural stem cells by defined factors","volume":"10","author":"Lee","year":"2012","journal-title":"Cell Stem Cell"},{"key":"29","doi-asserted-by":"crossref","first-page":"7838","DOI":"10.1073\/pnas.1103113108","article-title":"Direct reprogramming of mouse fibroblasts to neural progenitors","volume":"108","author":"Ding","year":"2011","journal-title":"Proc Natl Acad Sci U S A"},{"key":"30","doi-asserted-by":"crossref","first-page":"473","DOI":"10.1016\/j.stem.2012.03.003","article-title":"Direct conversion of fibroblasts into stably expandable neural stem cells","volume":"10","author":"Edenhofer","year":"2012","journal-title":"Cell Stem Cell"},{"key":"31","doi-asserted-by":"crossref","first-page":"100","DOI":"10.1016\/j.stem.2012.05.018","article-title":"Direct reprogramming of mouse and human fibroblasts into multipotent neural stem cells with a single factor","volume":"11","author":"Huang","year":"2012","journal-title":"Cell Stem Cell"},{"key":"32","article-title":"Rapid and efficient direct conversion of human adult somatic cells into neural stem cells by HMGA2\/let-7b","author":"Jung","year":"2015","journal-title":"Cell Rep"},{"key":"33","doi-asserted-by":"crossref","first-page":"528","DOI":"10.1042\/BST0360528","article-title":"Function and regulation of the mammalian Musashi mRNA translational regulator","volume":"36","author":"MacNicol","year":"2008","journal-title":"Biochem Soc Trans"},{"key":"34","doi-asserted-by":"crossref","first-page":"e50262","DOI":"10.1371\/journal.pone.0050262","article-title":"Sensitivity to lysosome-dependent cell death is directly regulated by lysosomal cholesterol content","volume":"7","author":"Kagedal","year":"2012","journal-title":"PLoS One"},{"key":"35","doi-asserted-by":"crossref","first-page":"477","DOI":"10.1007\/s11745-009-3305-7","article-title":"Cholesterol synthesis inhibitor U18666A and the role of sterol metabolism and trafficking in numerous pathophysiological processes","volume":"44","author":"Cenedella","year":"2009","journal-title":"Lipids"},{"key":"36","doi-asserted-by":"crossref","first-page":"6367","DOI":"10.1111\/febs.12505","article-title":"The potential of histone deacetylase inhibitors in Niemann - Pick type C disease","volume":"280","author":"Spiegel","year":"2013","journal-title":"FEBS J"},{"key":"37","first-page":"233","article-title":"Liver X receptor in cholesterol metabolism","volume":"204","author":"Dahlman-Wright","year":"2010","journal-title":"JEndocrinol"},{"key":"38","doi-asserted-by":"crossref","first-page":"1125","DOI":"10.1172\/JCI0215593","article-title":"SREBPs: activators of the complete program of cholesterol and fatty acid synthesis in the liver","volume":"109","author":"Brown","year":"2002","journal-title":"J Clin Invest"},{"key":"39","doi-asserted-by":"crossref","first-page":"20917","DOI":"10.1074\/jbc.R400040200","article-title":"Niemann-Pick type C disease and intracellular cholesterol trafficking","volume":"280","author":"Chang","year":"2005","journal-title":"J Biol Chem"},{"key":"40","doi-asserted-by":"crossref","first-page":"460","DOI":"10.1016\/j.scr.2016.02.025","article-title":"Induced neural stem cells from distinct genetic backgrounds exhibit different reprogramming status","volume":"16","author":"Ko","year":"2016","journal-title":"Stem Cell Res"},{"key":"41","doi-asserted-by":"crossref","first-page":"949","DOI":"10.1126\/science.1164270","article-title":"Generation of mouse induced pluripotent stem cells without viral vectors","volume":"322","author":"Yamanaka","year":"2008","journal-title":"Science"},{"key":"42","article-title":"Identification of NPC1 as the target of U18666A, an inhibitor of lysosomal cholesterol export and Ebola infection","volume":"4","author":"Brown","year":"2015","journal-title":"eLife"},{"key":"43","doi-asserted-by":"crossref","first-page":"16659","DOI":"10.1073\/pnas.0407643101","article-title":"Histone deacetylase inhibition-mediated neuronal differentiation of multipotent adult neural progenitor cells","volume":"101","author":"Gage","year":"2004","journal-title":"Proc Natl Acad Sci U S A"},{"key":"44","doi-asserted-by":"crossref","first-page":"473","DOI":"10.1016\/j.neuropharm.2008.09.019","article-title":"Valproic acid promotes neuronal differentiation by induction of proneural factors in association with H4 acetylation","volume":"56","author":"Son","year":"2009","journal-title":"Neuropharmacology"},{"key":"45","doi-asserted-by":"crossref","first-page":"941","DOI":"10.1038\/mp.2015.3","article-title":"Valproic acid enhances neuronal differentiation of sympathoadrenal progenitor cells","volume":"20","author":"Ehrhart-Bornstein","year":"2015","journal-title":"Mol Psychiatry"},{"key":"46","doi-asserted-by":"crossref","first-page":"569","DOI":"10.1517\/13543784.16.5.569","article-title":"Focus on acetylation: the role of histone deacetylase inhibitors in cancer therapy and beyond","volume":"16","author":"Papavassiliou","year":"2007","journal-title":"Expert Opin Invest Drugs"},{"key":"47","doi-asserted-by":"crossref","first-page":"4004","DOI":"10.1091\/mbc.e11-04-0372","article-title":"STARD4 abundance regulates sterol transport and sensing","volume":"22","author":"Maxfield","year":"2011","journal-title":"Mol Biol Cell"},{"key":"48","article-title":"Pitfalls in the detection of cholesterol in Huntington\u2019s disease models","volume":"4","author":"Cattaneo","year":"2012","journal-title":"PLoS Curr"}],"container-title":["Oncotarget"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/www.oncotarget.com\/lookup\/doi\/10.18632\/oncotarget.19976","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2022,7,31]],"date-time":"2022-07-31T21:07:27Z","timestamp":1659301647000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.oncotarget.com\/lookup\/doi\/10.18632\/oncotarget.19976"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2017,8,7]]},"references-count":48,"journal-issue":{"issue":"49","published-print":{"date-parts":[[2017,10,17]]}},"alternative-id":["19976","29156730"],"URL":"https:\/\/doi.org\/10.18632\/oncotarget.19976","relation":{},"ISSN":["1949-2553"],"issn-type":[{"value":"1949-2553","type":"electronic"}],"subject":[],"published":{"date-parts":[[2017,8,7]]}}}